FDA Approves Neurocrine Biosciences' Crenessity, First New Treatment for CAH in 70 Years

Dec 13, 2024, 08:13 PM

The U.S. Food and Drug Administration (FDA) has approved Neurocrine Biosciences' Crenessity (crinecerfont) for the treatment of adults and pediatric patients over 4 years of age with classic congenital adrenal hyperplasia (CAH). This approval marks the first new treatment option for CAH in 70 years, offering a paradigm-shifting approach by directly reducing excess adrenocorticotropic hormone (ACTH) and downstream adrenal androgen production, allowing for glucocorticoid dose reduction. The approval was supported by data from the largest-ever clinical trial program in pediatric and adult patients with classic CAH. Crenessity is expected to be commercially available in approximately one week.